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Gene therapy taking early steps into the cornea
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VIENNA, Austria — Gene therapy is already a reality for the retina, and it is moving into the early stages of potentially treating corneal diseases.
"The future looks promising. The ideas are there. We know how to modulate genes, and we have found that inactivated viral vectors can be our 'horse of Troy' also into the cornea," François Malecaze, MD, said at the EuCornea meeting here preceding the European Society of Cataract and Refractive Surgeons meeting.
Studies on the corneal epithelium are at a standstill because gene transfer has so far shown to be possible in vitro but not in vivo. As far as the stroma is concerned, recent publications have demonstrated that gene modulation is possible in animal models.
"We can deliver the virus by injection through a microneedle or by topical administration of eye drops containing the viral vector following epithelium removal," Dr. Malecaze said. "This opens new perspectives to treat corneal scarring and corneal pathologies secondary to gene mutations, such as corneal dystrophies. The deficient gene can be replaced with a healthy gene."
Concerning the endothelium, the idea is to optimize the cultured donor graft during organ culture by transfer of antiapoptotic genes in the endothelial cells.
"Ex vivo gene transfer will inhibit apoptosis, thus improving the quality of the donor before transplantation. You'll deliver to the recipient a very healthy graft, also avoiding the possible safety issues of in vivo gene therapy in patients," Dr. Malecaze said.
- Disclosure: Dr. Malecaze has no relevant financial disclosures.